The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
A gene therapy for retinal degenerative diseases receives an investigational new drug (IND) clearance from the FDA to move forward with the Phase 2 trial of the multi-characteristic opsin (MCO-010) therapy.
Since an effective treatment for the rare disease metachromatic leukodystrophy had not previously been distinguished, investigators analyzed data on the safety and efficacy of arsa-cel, a gene therapy.
The therapeutic met all secondary endpoints measured at 18 months in patients with hATTR amyloidosis with polyneuropathy, which included statistically significant improvements in neuropathy, and quality of life.
After conducting and extensive literary review on all eligible data regarding COVID-19 in patients with sickle cell disease or trait, experts discuss what is known and what needs to be better understood about the pandemic's effect on this population.